First Half 2018 Update

The CTSF had over 800 different donors in the first half of 2018. We raised $172,928. We had 21 fundraisers, with 14 of those done
on Facebook to mark a memory or special occasion. We had 11 Corporate matches and kind and generous folks donated to us from United Way contributions.

When the CTSF started, there basically was zippo in terms of research. Today, I talk regularly with leading researchers about Gene Therapy, Clinical Trials on real kids, FDA approvals, UK/and the European Union funding, Tanganil, and "Compassionate Care" laws. Yep, it's complicated. But the bottom line is that several research efforts are all coming together at once. So much will happen in the next 8-10 months. When I get the "OK" from several of the researchers that we have funded, you will be the first to know the details. All of this has happened because of your help.

We are too late for too many kids, including mine. But, as Churchill spoke to Parliament when it looked like the Nazi's would successfully invade, he said... "We shall never surrender". With your help, we won't either.

- Rick, and the CTSF Board

Cure Tay-Sachs Foundation

The Cure Is Within Reach!

Since our creation in June 2007, the CTSF has raised $4,456,711 from thousands of kind and generous donors. We have issued research grants to 9 researchers/laboratories around the world, and made impressive progress towards our ultimate goal... The Cure!

Tay-Sachs disease is caused by the absence or insufficient level of a vital enzyme called Hexosaminidase A (Hex-A).
Without Hex-A, a fatty substance or lipid called GM2 ganglioside accumulates abnormally in cells, especially in the nerve cells of
the brain. This ongoing accumulation, also called substrate, causes progressive damage to the cells. In Classic Infantile the
destructive process begins in the fetus early in pregnancy, although the disease is not clinically apparent (symptoms do not start)
until the child is several months old. By the time a child with Tay-Sachs disease is three or four-years old, the nervous system
is so badly affected that life itself cannot be supported. Even with the best of care, all children with classic Tay-Sachs disease
die early in childhood, usually by the age of 5, although some do live longer.

How do set up a free Facebook Fundraiser?

Many of you have asked how to set up a FB Fundraiser.... here ya go. You can create fundraisers for charitable organizations and personal causes on Facebook for Birthdays, Anniversary's, or any occasion!

IT'S FREE, IT'S FUN, AND ALL $$$ GOES TOWARDS RESEARCH

Tanganil: An easy way to share your experience...

Cure Tay-Sachs Foundation (CTSF) and NTSAD have collaborated with TREND Community to collect information from
families and individuals about their experiences using Tanganil, available without a prescription from Europe
and indicated for the treatment of positional vertigo (dizziness). It has not been proven to be effective or
approved for use to treat symptoms of GM1 and/or GM2 Tay-Sachs and Sandhoff.

There is no travel required to take part in this online community.

All data shared will be anonymous and de-identified.

The data will be used by researchers, doctors, and pharmaceutical companies to better understand how the disease impacts the family, generate ideas for future research and design better clinical trials.

Click the link below to download the TREND Community PDF to read more about this initiative, how it can help and how you can
sign up to be a part of this unique way to participate in research.

Here is the link. The instructions to sign up are right at the bottom.

"Holy Cow! Over 2000 people "Like" the CTSF Facebook page now, and our this website has been visited 6000
times in the last 12 months. I'm humbled! It's March 5th, and we have already brought in over $100,000
this year for research. With what we have planned for the rest of the year, you ain't seen nuttin yet!
Of course, we plan to write checks for over $800,000 this year. Our funding will see the first Gene Therapy
treatment this year, and two other clinical trials on affected children! There will be obstacles,
but there has never been..... not even close... this much hope. ALL our hope has been from your help. So,
if you have a rich crazy Uncle someplace, now is the time to send him our website address.
www.curetay-sachs.org.
Our work is never too late for our kids and those kids yet to be born.
They deserve a full and happy life. That's why we do this"

Family Stories

Nikko's Story

Nikko was born on May 14 2015 and he is our firstborn son.
Nikko since day one I always knew as his mother that he was angelic and
filled a room with so much love with just his presence.
What I didn't know was Nikko would be

Brennan's Story

Words cannot express what a privilege it was to be Brennan's parents.
The Lord chose us to care for this precious child and now he is home.
Brennan passed on March 4, 2016, in the comfort of his own home.
It was fairly sudden and our hearts are broken.
Brennan's parents, Holly and Royce Stringer, reside in Prescott, Arizona.

Alyssa's Story

Alyssa Tyner was born on a pretty morning on June 19, 2014. We were so happy and blessed to finally have our family complete. Alyssa was the little sister to Leilani, who felt proud to be the big loving sister.

Ryan's Story

Ryan Anthony Babo was born February 19, 2002. Like all Tay-Sachs children, he was absolutely beautiful. I remember lying in bed next to him when he was just a week old, watching him sleep, wondering what I had ever done in my life to deserve him.

Molly's Story

Elise's "Big Elise" Story

Elise Catherine ten Berge was born on February 5, 2003. She was our first child and as all new parents do, we thought about what Elise's future would be like: her first word, her first step, and her first day of school.

Dakota's Story

Dakota Jean Bihn was born on May 30, 1999. She was a beautiful baby, weighing in at exactly 8 pounds. Everything about her seemed perfect. During Dakota's first year, she developed as a normal baby should. She sat up, rolled over, ate, and walked, all when she should.

Conner's Story

Conner, a precious miracle and an amazing creature sent to Carl, Brenden and myself one cold, snowy January day. From the second he entered our life, I knew in my heart he was so incredibly special. His first smile, his first cry, even his first breath, he was--and remains--beautiful.

Lexi's Story

My name is Alexis Nichole Markowich or just "Lexi". I was born on March 9, 2001 to Craig and Nichole Markowich and I also have a big sister Brianne. When I was four, on August 4, 2005, we got the devastating news that I had Juvenile Onset Tay Sachs Disease (JOTSD).

Isabel's Story

Isabel was born in Buenos Aires on February of 2009. Until the age of two Isabel was a healthy little girl. She had the life that any other child could have: school, little friends at park, holidays and trips to Spain to visit her family. And always a big smile on her face...

Lexi's Story

Nathan's Story

Our son Nathan Harney is almost 17 months old. He is a wonderful, happy baby who has beautiful blue eyes, a huge personality, and is the light of our lives. He is a gentle soul that we have been blessed with......

Isaiah's Story

Rachaeli's Story

Rachaeli entered this world with her eyes open wide; God delivered her to us shortly before the start of the Sabbath on Friday October 25, 2002. It was a perfect delivery, following a perfect pregnancy. Rachaeli was absolutely captivating.

Kaiden's Story

Kaiden was born December 20, 2006. He was my beautiful blue eye, blond hair angel. He was the best baby any parents could ask for. He smiled constantly and barely cried. I would tell my Mother all the time that he was my "special little man".......

Elise's "Baby Elise" Story

Elise was born December 1, 2004 in Santa Maria, California. She was an absolutely perfect baby with an easy pregnancy. We never imagined we could love someone as much as we love Elise. The next 6 months were the happiest months of our lives......

Cole's Story

Travis Cole Snellgrove was born to Wendi and Travis on April 12th, 2004. He was born with no problems and was home the next day. He was a very good baby and we had no problems with him for the first month, then he started having trouble.....

DJ's Story

Dylan James Manning (commonly referred to as "DJ") was born on Thanksgiving Day, November 22, 2001. Dylans arrival was a long awaited event for his parents, Brian and Sherri of Atglen, Pennsylvania. Although Dylan was anxious to arrive almost four weeks early, he was not quick to reach his milestones. Doctors naturally gave him a little extra time to reach things like crawling and rolling over due to his prematurity.

Amelia's Story

Amelia was born September 29, 2006 and was a sweet, easy-going baby right from the start. Her baby book filled up as it should have first smile at four weeks, rolled over at four months, babbled at five months.

Emma's Story

Lacie's Story

My name is Lacie Wivell and this is my story. I was born on April 8, 2004, to Rick and Lisa Wivell. I had two older sisters, Amanda and Rikki, and two older brothers, Levi and Colby. I also had another brother, Dakota, that went to be an angel in 2001.

Gene therapy for Tay-Sachs and Sandhoff diseases has been redesigned to reduce the potency of the vectors, thereby increasing safety. Studies have been conducted with guidance from the FDA about what is needed for clinical trials to begin.

The new vector system clearly restores hexosaminidase activity after injection into the brains of mice, and storage levels are decreased. Treated mice have increased life spans and show no side effects of the treatment.

To thoroughly test safety, the new vector system has been tested in non-human primates (monkeys) for almost 1 year, with no evidence of toxicity on MRIs or behavioral tests. A few steps remain before the clinical trial can begin: monkey tissues need to be analyzed microscopically by pathologists, large-scale vector manufacturing must be completed, and regulatory documents must be prepared and filed. All of these activities can begin when funding is available.

From: Dr. Joe Anderson The University of California - Davis

Currently we are continuing our in vivo efficacy and safety studies. We have observed a significant decrease in GM2 aggregates in the brains of mice engrafted with a high percentage of human cells transduced with the HexA/HexB lentiviral vector.

We have also seen an extension of lifespan in these mice. Human hematopoietic stem cells transduced with the HexA/HexB lentiviral vector also matured into normal immune system cells when engrafted in a humanized mouse model. We are repeating some of these experiments so that we have enough mice to get statistically significant data.

Once these experiments are completed, we will be able to have a pre-IND meeting with the FDA.

"One in a Million"

The Cure Tay-Sachs Foundation is very grateful you decided to donate and we thank you for your generosity. Your donation will be used to support research to find a cure for Tay-Sachs disease.

The Cure Tay-Sachs Foundation is exempt under Section 501(c)(3) of the Internal Revenue Code, making this gift tax deductible to the fullest extend permitted by law.

Please click the PayPal button to make a donation today! You can donate using a credit card - without a paypal account - by following the instruction on the paypal system (lower left). Or, if you prefer, please send your donation (payable to the Cure Tay-Sachs Foundation) to the address below.